Beyond Achondroplasia

Growing together with Clara

BMN-111 phase 2 results presented at the ASBMR 2015 meeting


Dr. Melita Irving, the clinical geneticist who is coordinating the phase 2 clinical trial of BMN-111 in London, presented at the American Society for Bone and Mineral Research Annual 2015 Meeting (ASBMR) the initial six-month data from the first three cohorts of a Phase 2 proof-of-concept and dose-finding study of vosoritide (BMN 111).

This is not new. These results have been already disclosed in June 2015. You can read the post here. And this press release can be read here.

In the excerpt of the press release on the 12th October:

The initial six-month data from the first three cohorts showed a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (p-value= 0.01). Data suggests that vosoritide activity was sustained over six months of dosing as measured by increases in cyclic guanosine monophosphate (cGMP), a urinary marker of pharmacological activity (biomarker)

Biomarker: Is a measurable and quantifiable biological parameter (e.g., specific enzyme concentration, specific hormone concentration, specific gene phenotype distribution in a population, presence of biological substances) which serve as indices for health and physiology related assessments.

No serious or severe adverse events were observed and the most common adverse events reported were mild injection site reactions, asymptomatic hypotension and headache that were resolved without medical intervention (Grade 1).

Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer at BioMarin said: “We are pleased to share this initial six-month data from the first three cohorts in a scientific forum, and we are grateful to the children, families and physicians who have participated in this study. By developing a therapy that addresses the root cause of achondroplasia, we hope to address the associated complications, such as disproportionate bone growth,” . “We are very encouraged with this initial data, and we look forward to working with health authorities and the patient community to advance vosoritide to the next stage of clinical development.”


While the Phase 2 efficacy endpoint centers on annualized growth velocity after six months of treatment, BioMarin believes that growth velocity may be an important early indicator and that longer treatment may lead to improvement in many of the complications that can be associated with achondroplasia, such as disproportionality, though longer time of treatment is likely required to accumulate evidence to this effect.

This is probably one of the biggest ambitions of BioMarin and all of us: the desired positive outcome related to the cumulative use (daily administration since early age until puberty) of Vosoritide: recovering the proportionality (limbs to torso) and decreasing the anatomic alterations that occur in achondroplasia that originate several complications such as the severely reduced growth (small widening) of the foramen magnum.

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